1. In two clinical trials, iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH) improved hemoglobin levels compared to anti-C5-treated patients and those without complement inhibitors. 2. Patients who received iptacopan generally exhibited improved secondary outcomes, including less fatigue, reduced reticulocyte and bilirubin levels, and decreased breakthrough hemolysis. Evidence Rating Level: 1 (Excellent) Study Rundown: PNH is a
1. In two clinical trials, iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH) improved hemoglobin levels compared to anti-C5-treated patients and those without complement inhibitors. 2. Patients who received iptacopan generally exhibited improved secondary outcomes, including less fatigue, reduced reticulocyte and bilirubin levels, and decreased breakthrough hemolysis. Evidence Rating Level: 1 (Excellent) Study Rundown: PNH
Results showed treatment with iptacopan through 48 weeks caused hemoglobin-level increases, blood transfusion avoidance, and reductions in patient-reported fatigue.
Novartis (NVS) reported positive topline results from the six-month, double-blind period of the Phase III APPEAR-C3G study of iptacopan for the treatment of patients with C3 glomerulopathy.